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2007;7(2):1319. Epub 2013 Apr 16. Bagley SJ, et al. Adult T-cell leukemia/lymphoma (ATL) is an aggressive non-Hodgkin lymphoma with poor prognosis and few treatment options for patients with relapsed, recurrent, or refractory disease. Pharm Stat. Patients will receive up to 18 IT cycles after enrollment. And the values of two types of error must be clearly stated in the protocol to assess the certainty of the results and the power of the study. 2022 Dec 6;15:1481-1501. doi: 10.2147/OTT.S326632. 2022 Jul 21;17(7):e0271094. Multicenter Phase II Study of Lenalidomide in Relapsed or Recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002. A Phase 2 single-arm study using two-stage design [42], where a new treatment option of hypoxia activated evofosfamide (TH302) for patient with recurrent bevacizumab-refractory glioblastoma was investigated. If equal to or more than 2 of these patients are progression-free after 4months, the trial continues with additional 22 patients. Findings: Between March 30, 2015, and Aug 3, 2018, 79 patients were enrolled. A phase 2 study of the first imipridone ONC201, a selective DRD2 antagonist for oncology, administered every three weeks in recurrent glioblastoma. Patients pretreated with mogamulizumab had an ORR of 45.8% (4 complete and 7 partial remissions). If a HR is used along with a one-sample log-rank test, the required sample size becomes 47, 43, and 40 patients when the expected accrual duration is 6, 12, and 24months, respectively. Wouters and colleagues reported that U. S. biopharmaceutical companies spent approximately $1 billion to bring each new drug to market between 2009 and 2018. But interestingly almost 60% of studies (17/29, 59%) failed to provide at least one key output results of sample size calculation such as the number of samples of first stage and both stages (\({n}_{1}, n\)) and the treatment rejection numbers of the first stage and both stages (\({r}_{1}, r\)). eCollection 2020 Dec. BMC Med Res Methodol. 2016 Dec;34(34):4086-4093. doi: 10.1200/JCO.2016.67.7732. Patients pretreated with mogamulizumab had an ORR of 45.8% (4 complete and 7 partial remissions). Combining progression-free survival and overall survival as a novel composite endpoint for glioblastoma trials. 2019;144(2):35968. Careers. Please check for further notifications by email. Atorvastatin in combination with radiotherapy and temozolomide for glioblastoma: a prospective phase II study. 2011;103(3):491501. 2013;24(4):11129. An Open-Label, Single-Arm, Phase 2 Trial of Valemetostat in Relapsed or Refractory Adult T-Cell Leukemia/Lymphoma - ScienceDirect Available online 27 Expert Rev Anticancer Ther. Most trials (n=22, 76%) successfully provided all key information while 7 trials (24%) failed to provide at least one key information (Six trials failed to provide two types of error rates, four trials failed to provide two response rates, and three trials failed to provide two or more than two key results from the sample size calculation). WebDive into the research topics of 'MATRix-RICE therapy and autologous haematopoietic stem-cell transplantation in diffuse large B-cell lymphoma with secondary CNS involvement (MARIETTA): an international, single-arm, phase 2 trial'. For the primary endpoint of 4-month progression-free survival (PFS4), an uninteresting rate of 10.9% obtained from historical controls and an anticipated rate of 28.9% were assumed. eCollection 2023 Feb. Tannoury M, Garnier D, Susin SA, Bauvois B. This systematic review has some limitations. Optimal, minimax and admissible two-stage design for phase II oncology clinical trials. 2018;7(4):e1412901. We would like to thank the editors and anonymous reviewers for all valuable comments and suggestions, which helped us to improve the quality of the manuscript. 2015;34(6):1031-1040. An International, Single-Arm, Multicenter Phase 2 Trial. Friends of Cancer Research is a 501 (C)(3) non-profit organization.Our tax ID number is 52-1983273. Figure2 summarized frequencies and proportions from identified ten topics related Phase 2 single-arm two-stage designs: (1) disease (Yes: GBM, No: glioma), (2) setting (Yes: recurrent, No: newly-diagnosed), (3) patients (Yes: adults, No: child), (4) therapeutic drug (Yes: single, No: combination), (5) primary endpoint (Yes: PFS6, No: ORR and others), (6) methods of two-stage sign (Yes: Simon, No: others), (7) all four key input information of two-stage design provided? 2022 Dec;17(6):524-532. doi: 10.1159/000526431. Patients received valemetostat 200 mg/day until progressive disease or unacceptable toxicity. 2019;142(3):45562. HHS Vulnerability Disclosure, Help Taylor JW, et al. Invest New Drugs. Among examined 29 trials, 12 trials (41%) appropriately provided key input parameters and sample size results from two-stage design implementation. doi: 10.1016/S2352-3026(22)00072-2. There has been the need for new two-stage designs that allow flexible modification of design parameters under the control of the Type 1 error, which is called adaptive design methods to perform arbitrary design modification under the control of the Type 1 error rate. 2021;13(3):28694. 2021;150:5362. 7 Briefly, SAUL ( NCT02928406) was a single-arm phase b study of atezolizumab in a real-world patient population with locally advanced or metastatic UC or non-UC of the urinary tract. DU reports personal fees from ADC Therapeutics, during the conduct of the study; and has a patent (20200171164) pending. Fifty-six of the 70 (80%) participants became desensitized to peanuts. Epub 2019 Nov 26. Cancer immunotherapy trial design with long-term survivors. The Phase 2 trials often apply single-arm study designs to identify the efficacious treatment by using historical control data for comparative evaluation with study treatment data. Disclaimer, National Library of Medicine 2021 Dec;21(12):1313-1320. doi: 10.1080/14737140.2021.1988853. The early phase of trials might encounter a considerable amount of uncertainty when planning a trial. For general information, Learn About Clinical Studies. 2013;33(4):165760. Contemp Clin Trials. Pembrolizumab is safe and feasible and displays promising activity in patients with LMD. This site needs JavaScript to work properly. Accessibility Evaluation of two-stage designs of Phase 2 single-arm trials in glioblastoma: a systematic review, https://doi.org/10.1186/s12874-022-01810-7, Two-stage design of phase 2 single-arm trials in glioblastoma, http://creativecommons.org/licenses/by/4.0/, http://creativecommons.org/publicdomain/zero/1.0/, bmcmedicalresearchmethodology@biomedcentral.com. Gehan EA. Intrathecal administration of trastuzumab for the treatment of meningeal carcinomatosis in HER2-positive metastatic breast cancer: a systematic review and pooled analysis. 2015;121(2):297302. Lassen U, et al. Epub 2020 Jun 5. Please enable it to take advantage of the complete set of features! Loss of power according to the underestimation rate of a null hypothesis (p0) when a single-arm single-stage design is used for a binary endpoint Under 80% of power and 5% of one-sided Type 1 error rate. doi: https://doi.org/10.1182/blood.2022016862. Polatuzumab vedotin plus obinutuzumab and lenalidomide in patients with relapsed or refractory follicular lymphoma: a cohort of a multicentre, single-arm, phase 1b/2 study. Zhou H, Lee JJ, Yuan Y. BOP2: Bayesian optimal design for phase II clinical trials with simple and complex endpoints. Anand S, et al. 2015;17(8):110613. and transmitted securely. J Nat Cancer Inst. 2006;25(19):338295. Neuro Oncol. FOIA J Cancer. 2022 Oct;23(10):1457-1476. doi: 10.1007/s11864-022-01011-w. Epub 2022 Sep 22. Sanai N. Phase 0 clinical trial strategies for the neurosurgical oncologist. Careers. Nat Commun. -, Ongerboer de Visser, B. W. et al. Current Status of Novel Agents for the Treatment of B Cell Malignancies: What's Coming Next? 2016;43(1):138. PubMedGoogle Scholar. This shows that oncology clinical trials have the lowest success rate on Phase 2 to Phase 3 trials and the highest median expense in a new drug to market, compared to other disease areas. We conducted a single-arm, sharing sensitive information, make sure youre on a federal This site needs JavaScript to work properly. Disclaimer, National Library of Medicine Federal government websites often end in .gov or .mil. 2022 Dec 26;56:101779. doi: 10.1016/j.eclinm.2022.101779. The optimal historical control success rates depend on the number of historical data, variability in historical success rates, patient selection differences. Would you like email updates of new search results? The followings were key words for the literature search as index terms or free-text words: phase II trials, glioblastoma, and two-stage design. 2003;95(19):1434-1439. J Neurooncol. 2012;106(3):6439. 2017;131(3):60310. Estimation of clinical trial success rates and related parameters. Overly optimistic results obtained from Phase 2 trials might be a major reason of negative Phase 3 results in randomized studies. Keywords: doi: 10.1371/journal.pone.0246448. Stat Med. Only around 41% of trials (n=12) appropriately provided the key input and output information for the study design and sample size calculation of two-stage designs phase 2 trials. We show that Simon's two-stage designs for efficacy studies can be similarly used to design a two-stage safety study by modifying some of the design parameters. WY, SK, and MG wrote the main manuscript text and WY and SK prepared Table 1 and Figs. Eltarhoni K, Kamel F, Ihebunezie K, Nisar P, Soloviev M. Int J Mol Sci. Thus, the maximum unacceptable response rates for historical controls should be considered throughout literature examination and/or previous research experience to screen out the inefficacious treatments [47, 48]. The https:// ensures that you are connecting to the Phase II open-label study of nintedanib in patients with recurrent glioblastoma multiforme. Epub 2021 May 11. There is no universal solution to handle the variability around historical control data since the selected design depends on judgements to the specific study circumstances. Unable to load your collection due to an error, Unable to load your delegates due to an error. Zhou H, et al. Ostrom QT, et al. A recent systematic review shows that both RR and PFS6 are suitable surrogate endpoints for OS, but their surrogacy varies according to therapy line or type and study size [53]. Phase 3 results in randomized studies: a prospective Phase II clinical trials progressive disease or unacceptable toxicity input. ( 10 ):1457-1476. doi: 10.1159/000526431 -, Ongerboer de Visser, W.... Reports personal fees from ADC Therapeutics, during the conduct of the study ; and has a (. Work properly metastatic breast Cancer: a systematic review and pooled analysis ATLL-002., B. W. et al after enrollment, Garnier D, Susin SA, B! ):4086-4093. doi: 10.1007/s11864-022-01011-w. 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